Efficacy of bezafibrate for preventing myopathic attacks in patients with very long-chain acyl-CoA dehydrogenase deficiency.

Abstract

BACKGROUND:

Very long-chain acyl-CoA dehydrogenase deficiency (VLCADD) is a mitochondrial fatty acid oxidation disorder that causes episodic attacks, such as general fatigue, hypotonia, myalgia, and rhabdomyolysis accompanied by lack of energy. As yet, there are no preventative drugs for these VLCADD-associated metabolic attacks.

PATIENTS AND METHODS:

We conducted an open-label, non-randomized, multi-center study into the effects of bezafibrate on five patients with VLCADD. Bezafibrate was administered for 4 years, and we analyzed the number of myopathic attacks requiring hospitalization and treatment infusions.

RESULTS:

The number of myopathic attacks requiring infusions of 24 h or longer significantly decreased during the study period. The patients' ability to conduct everyday activities was also improved by the treatment.

CONCLUSION:

Our findings show the potential long-term efficacy of bezafibrate in preventing myopathic attacks for patients with VLCADD.

• Authors: Hideaki Shiraishi, Kenji Yamada, Kiyoshi Egawa, Mika Ishige, Fumihiro Ochi, Asami Watanabe, Sanae Kawakami, Kazuyo Kuzume, Kenji Watanabe, Koji Sameshima, Kiyotaka Nakamagoe, Akira Tamaoka, Naoko Asahina, Saki Yokoshiki, Keiko Kobayashi, Takashi Miyakoshi, Koji Oba, Toshiyuki Isoe, Hiroshi Hayashi, Seiji Yamaguchi, Norihiro Sato
• Journal: Brain & development (Brain Dev) Vol. 43 Issue 2 Pg. 214-219 (Feb 2021) ISSN: 1872-7131 [Electronic] Netherlands
• PMID: 32798077 (Publication Type: Clinical Study, Journal Article, Multicenter Study)
• Copyright: Copyright © 2020 The Japanese Society of Child Neurology. Published by Elsevier B.V. All rights reserved.

Chemical References

• Acyl-CoA Dehydrogenase, Long-Chain
• Bezafibrate

Topics

• Acyl-CoA Dehydrogenase, Long-Chain (deficiency, genetics)
• Adult
• Bezafibrate (metabolism, therapeutic use)
• Child
• Congenital Bone Marrow Failure Syndromes (drug therapy, physiopathology)
• Female
• Humans
• Lipid Metabolism, Inborn Errors (drug therapy, physiopathology)
• Male
• Mitochondrial Diseases (drug therapy, physiopathology)
• Muscular Diseases (drug therapy, physiopathology)